Prospective multicenter trial comparing repeated immunosuppressive therapy with stem-cell transplantation from an alternative donor as second-line treatment for children with severe and very severe aplastic anemia

Yoshiyuki Kosaka, Hiroshi Yagasaki, Kimihiko Sano, Ryoji Kobayashi, Hiroshi Ayukawa, Takashi Kaneko, Hiromasa Yabe, Masahiro Tsuchida, Hideo Mugishima, Akira Ohara, Akira Morimoto, Yoshitoshi Otsuka, Shouichi Ohga, Fumio Bessho, Tatsutoshi Nakahata, Ichiro Tsukimoto, Seiji Kojima

研究成果: Contribution to journalArticle査読

79 被引用数 (Scopus)

抄録

We conducted a prospective multicenter study to compare the efficacy of repeated immunosuppressive therapy (IST) with stem-cell transplantation (SCT) from an alternative donor in children with acquired aplastic anemia (AA) who failed to respond to an initial course of IST. Patients with severe (n = 86) and very severe disease (n = 119) received initial IST consisting of antithymocyte globulin (ATG) and cyclosporine. Sixty patients failed to respond to IST after 6 months from the initial IST and were eligible for second-line treatment. Among them, 21 patients lacking suitable donors received a second course of IST. Three patients developed an anaphylactoid reaction to ATG and could not complete the second IST. A trilineage response was seen in only 2 of 18 (11%) evaluable patients after 6 months. Thirty-one patients received SCT from an alternative donor. At 5 years from the initiation of second-line therapy, the estimated failure-free survival (FFS), defined as survival with response, was 83.9% (± 16.1%, SD) in the SCT group compared with 9.5% (± 9.0%) in the IST group (P = .001). These results suggest that SCT from an alternative donor offers a better chance of FFS than a second IST in patients not responding to an initial IST.

本文言語英語
ページ(範囲)1054-1059
ページ数6
ジャーナルBlood
111
3
DOI
出版ステータス出版済み - 2008
外部発表はい

All Science Journal Classification (ASJC) codes

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology

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